Actress Farrah Fawcett was first diagnosed with anal cancer in 2006. She died in 2009 at the age of 62. Photo credit: rocor/Creative Commons

Immunotherapy developer Advaxis is angling to carve out a space in the cancer market relating to human papillomavirus. The FDA awarded axalimogene filolisbac (AXAL) with orphan designations for anal cancer as well as invasive cervical and head and neck squamous cell cancers.

“Axalimogene filolisbac is emerging as an exciting potential immunotherapy to treat HPV-associated cancers, of which there are many types of cancer with far too few treatment options,” says Advaxis CEO Daniel O'Connor.

Having recently overcome an FDA roadblock, the company's lead agent appears to be on solid footing again. The FDA put the firm's mid-stage trials on hold after a patient died, despite company insistence that the event resulted from disease progression. The FDA lifted the hold a few months later, with Advaxis agreeing to implement a few risk-mitigation measures.

In addition to leaving trial participants in limbo, the hold caused a blow to the firm's financial profile. Company shares went on a wild roller-coaster ride both during and after the hold. “The removal of the clinical hold allows us to expand our AXAL clinical programs and begin to move into even more HPV cancer and precancer indications,” O'Connor notes.

The number of new anal-cancer cases is on the rise, with about 7,270 new diagnoses in 2014, according to the American Cancer Society. About one in 500 people are at risk of an anal-cancer diagnosis; nearly all cases are HPV-related. The social stigma that burdens anal cancer, the HPV and Anal Cancer Foundation notes, prevents the proper evolution of awareness and funding to support research and care for people with the disease.

Advaxis develops its immunotherapies based on a technology platform using engineered listeria monocytogenes, licensed from the University of Pennsylvania. The novel approach adds another tool to the oncologist's armamentarium, one that may be more effective and less toxic than traditional cancer drugs.

Advaxis is enrolling patients for its second Phase II clinical study in patients with metastatic anal cancer. The two-part single-arm open-label study, called FAWCETT (Fighting Anal Cancer with CTL Enhancing Tumor Therapy) in honor of victim Farrah Fawcett, will evaluate the efficacy and safety of AXAL as monotherapy in patients who have received at least one prior treatment regimen for advanced disease. The study, in collaboration with Brown University Oncology Research Group, will evaluate whether AXAL will increase response rates when added to the standard care treatment of chemotherapy and radiation.

Throughout 2016, Advaxis plans to expand its collaborations with academic institutions and other companies to develop new combination regimens. “We appreciate the FDA's action, as we're working in multiple orphan indications where there's significant unmet need,” O'Connor acknowledges.